Potential Alzheimer’s Breakthrough: Cancer Drugs May Reverse Brain Damage

In a significant scientific breakthrough, researchers from UC San Francisco and Gladstone Institutes have identified two FDA-approved cancer drugs with the potential to reverse the devastating effects of Alzheimer’s disease on the brain. The study, published in Cell on July 21, 2025, revealed that letrozole (a breast cancer medication) and irinotecan (used for colon and lung cancer) could counteract disease-related changes in neurons and other brain cells.

The research team utilized computational tools to sift through over 1,300 drugs, searching for those that caused changes in gene expression opposite to those seen in Alzheimer’s disease. When tested in mouse models, the combination of letrozole and irinotecan not only reduced brain degeneration but also restored memory function.

Further supporting their findings, the researchers analyzed millions of electronic medical records. They discovered that patients who had taken these medications for other conditions were less likely to develop Alzheimer’s disease over time.

“We’re excited that our computational approach led us to a potential combination therapy for Alzheimer’s based on existing FDA-approved medications,” said co-senior author Marina Sirota, PhD, interim director of the UCSF Bakar Computational Health Sciences Institute.

Source: ScienceDaily

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